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Last update 08 May 2025

Calcitriol

Last update 08 May 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

(1S,3R,5Z,7E)-9,10-secocholesta-5,7,10-triene-1,3,25-triol, (1α,3β,5Z,7E)-9,10-secocholesta-5,7,10(19)-triene-1,3,25-triol, (5Z,7E)-(1S,3R)-9,10-secocholesta-5,7,10(19)-triene-1,3,25-triol

+ [24]

Target

VDR

Action

agonists

Mechanism

VDR agonists(Vitamin D receptor agonists)

Therapeutic Areas

Immune System DiseasesEndocrinology and Metabolic DiseaseSkin and Musculoskeletal Diseases+ [1]

Active Indication

Plaque psoriasisGlucocorticoid-induced osteoporosisHypocalcemia+ [7]

Inactive Indication

Advanced cancerAdvanced Lung Non-Small Cell CarcinomaCastration-Resistant Prostatic Cancer+ [8]

Originator Organization

Validus Capital Pte Ltd.

Active Organization

Esjay Pharma LLC

Hainan Sihuan Pharmaceutical Co. Ltd.

Kyowa Kirin Co., Ltd.

+ [5]

Inactive Organization

Zhejiang Cheng Yi Pharmaceutical Co., Ltd.

Novacea, Inc.

Galderma Research & Development SNC

+ [1]

License Organization

Fosun Wanbang (Jiangsu) Pharmaceutical Group Co., Ltd.

Rockwell Medical, Inc.

Drug Highest PhaseApproved

First Approval Date

United States (17 Aug 1978),

Hyperparathyroidism, SecondaryKidney Failure, Chronic

Regulation-

Structure/Sequence

Molecular FormulaC27H44O3

InChIKeyGMRQFYUYWCNGIN-NKMMMXOESA-N

CAS Registry32222-06-3

203

Clinical Trials associated with Calcitriol

CTR20243049

/ CompletedNot Applicable

骨化三醇软胶囊在健康人体空腹/餐后状态下的生物等效性试验

[Translation] Bioequivalence study of calcitriol soft capsules in healthy volunteers under fasting/fed conditions

主要研究目的：本研究以珠海健福制药有限公司持证的骨化三醇软胶囊（规格：0.25μg）为受试制剂，Atnahs Pharma UK Limited 持证的骨化三醇胶丸（商品名：罗盖全®，规格：0.25μg）为参比制剂，进行空腹/餐后状态下人体生物等效性试验，评价受试制剂和参比制剂在健康受试者体内的生物等效性。次要研究目的：观察受试制剂和参比制剂在健康受试者中的安全性。

[Translation]

Main research purpose: This study uses the calcitriol soft capsules (specification: 0.25μg) certified by Zhuhai Jianfu Pharmaceutical Co., Ltd. as the test preparation and the calcitriol capsules (trade name: Luogaiquan®, specification: 0.25μg) certified by Atnahs Pharma UK Limited as the reference preparation to conduct a human bioequivalence test under fasting/postprandial conditions to evaluate the bioequivalence of the test preparation and the reference preparation in healthy subjects. Secondary research purpose: Observe the safety of the test preparation and the reference preparation in healthy subjects.

Start Date14 Sep 2024

Sponsor / Collaborator

Zhuhai Jianfu Pharmaceutical Co., Ltd.

CTR20242334

/ CompletedNot Applicable

骨化三醇胶丸在中国健康受试者中的一项随机、开放、空腹和餐后单次给药、两制剂、两序列、两周期交叉设计的生物等效性试验

[Translation] A randomized, open-label, single-dose, fasting and fed, two-formulation, two-sequence, two-period crossover bioequivalence study of calcitriol softgels in healthy Chinese subjects

主要研究目的研究空腹和餐后状态下单次口服骨化三醇胶丸（规格：0.25μg，大连美创药业有限公司）与参比制剂（规格：0.25μg，罗盖全®；持证商：Roche Pharma (Schweiz) AG）后骨化三醇在中国健康受试者体内的药代动力学（PK）特征，评价空腹和餐后状态下口服两种制剂的生物等效性。次要研究目的评价中国健康受试者空腹和餐后状态下，单次口服受试制剂骨化三醇胶丸和参比制剂骨化三醇胶丸后的安全性。

[Translation]

Main study objectives To study the pharmacokinetic (PK) characteristics of calcitriol in healthy Chinese subjects after a single oral administration of calcitriol capsules (specification: 0.25μg, Dalian Meichuang Pharmaceutical Co., Ltd.) and reference preparation (specification: 0.25μg, Rogaquan®; licensee: Roche Pharma (Schweiz) AG) in fasting and fed states, and to evaluate the bioequivalence of the two preparations in fasting and fed states. Secondary study objectives To evaluate the safety of the test preparation calcitriol capsules and reference preparation calcitriol capsules in healthy Chinese subjects after a single oral administration in fasting and fed states.

Start Date12 Jul 2024

Sponsor / Collaborator

Dalian Meichuang Pharmacy Co., Ltd.

CTR20240267

/ CompletedNot Applicable

骨化三醇胶丸在中国健康受试者中空腹和餐后状态下的随机、开放、两序列、两周期交叉设计生物等效性试验

[Translation] A randomized, open-label, two-sequence, two-period crossover bioequivalence study of calcitriol softgels in Chinese healthy subjects under fasting and fed conditions

主要研究目的研究空腹和餐后状态下单次口服骨化三醇胶丸（规格：0.25μg，山东简道制药有限公司）与参比制剂（规格：0.25μg，罗盖全®；持证商：Roche Pharma (Schweiz) AG）后骨化三醇在中国健康受试者体内的药代动力学（PK）特征，评价空腹和餐后状态下口服两种制剂的生物等效性。次要研究目的评价中国健康受试者空腹和餐后状态下，单次口服受试制剂骨化三醇胶丸和参比制剂骨化三醇胶丸后的安全性。

[Translation]

Main study objectives To study the pharmacokinetic (PK) characteristics of calcitriol in healthy Chinese subjects after a single oral administration of calcitriol capsules (specification: 0.25μg, Shandong Jiandao Pharmaceutical Co., Ltd.) and reference preparation (specification: 0.25μg, Rogaquan®; licensee: Roche Pharma (Schweiz) AG) in fasting and fed states, and to evaluate the bioequivalence of the two preparations in fasting and fed states. Secondary study objectives To evaluate the safety of the test preparation calcitriol capsules and reference preparation calcitriol capsules in healthy Chinese subjects after a single oral administration in fasting and fed states.

Start Date20 May 2024

Sponsor / Collaborator

Shandong Jiandao Pharmaceutical Co., Ltd.

100 Clinical Results associated with Calcitriol

100 Translational Medicine associated with Calcitriol

100 Patents (Medical) associated with Calcitriol

23,857

Literatures (Medical) associated with Calcitriol

31 Dec 2025·Future Science OA

Harnessing tomato-derived small extracellular vesicles as drug delivery system for cancer therapy

Article

Author: Sarwareddy, Kartik Kumar ; Singh, Anula Divyash ; Manda, Venkata Sasidhar ; Sesuraj, Babiola Annes ; Thakur, Basant Kumar ; Patnam, Sreekanth ; Ponamgi, spd

AIMThis study aims to explore a sustainable and scalable approach using tomato fruit-derived sEVs (TsEVs) to deliver calcitriol for enhanced anticancer effects, addressing challenges of low yield and high costs associated with mammalian cell-derived sEVs.METHODSTsEVs were isolated by centrifugation and ultrafiltration and characterized using DLS, TEM, and biochemical assays. Calcitriol was loaded into TsEVs via loading methods, with efficiency measured by spectrophotometry and HPLC. HCT116 and HT29 colon cancer cells were treated with TsEV-calcitriol and assessed for viability, colony formation, migration, ROS levels, and apoptosis gene expression.RESULTSIsolated TsEVs ranged from 30-200 nm with a protein-to-lipid ratio of ∼1. Calcitriol encapsulation efficiencies were 15.4% (passive), 34.8% (freeze-thaw), and 47.3% (sonication). TsEV-calcitriol reduced HCT116 cell viability with IC50 values of 4.05 µg/ml (24 h) and 2.07 µg/ml (48 h). Clonogenic assays showed reduced colony formation and migration. Elevated ROS levels and increased Bax/Bcl-2 ratio were observed in treated HCT116 and HT29 colon cancer cells.CONCLUSIONThese findings highlight TsEVs as a promising alternative drug delivery platform to mammalian cell-derived sEV for enhancing the therapeutic efficiency of calcitriol and other anticancer agents.

01 Dec 2025·Molecular Biology Reports

A short review: the biological activity of vitamin D and its decomposition products

Review

Author: Shimomura, Hirofumi ; Hosoda, Kouich ; Amgalanbaatar, Avarzed ; Hayashi, Shunji ; Wanibuchi, Kiyofumi ; Shoji, Mitsuru

A vitamin D₃ metabolite 1α,25-dihydroxyvitamin D₃ exhibits numerous hormonal actions to various tissues and cells such as induction of calcium ion absorption, bone metabolism, activation of innate immune system. Because hormonal actions of 1α,25-dihydroxyvitamin D₃ are too complex, it is difficult to better understand overall views of intermolecular networks on the cell activation by 1α,25-dihydroxyvitamin D₃. In this review we concisely outline the most fundamental molecular mechanisms for the cell activation by 1α,25-dihydroxyvitamin D₃ based on the previous and recent studies. In addition, we expound biological activity of 25-hydroxyvitamin D₃ and 1α,25-dihydroxyvitamin D₃ that differs from hormonal actions. In addition, recent studies by our group revealed that vitamin D decomposition products confer extremely selective bactericidal action to Helicobacter pylori, a pathogen causative gastritis, peptic ulcers and gastric cancers in human. Vitamin D decomposition products induce the bacteriolysis as targeting of characteristic glycerophospholipids composed of the bacterial biomembranes. In the future we will expect to be capable of developing novel antibacterial agents targeting a characteristic glycerophospholipid of the pathogenic bacteria using vitamin D decomposition products as fundamental structures.

01 Dec 2025·Inflammation Research

Vitamin D3 suppresses NLRP3 inflammasome pathway and enhances steroid sensitivity in a neutrophilic steroid hyporesponsive asthma mouse model

Article

Author: Khalil, Bariaa ; Selvakumar, Balachandar ; Mdkhana, Bushra ; Zakri, Adel ; Hachim, Ibrahim ; Halwani, Rabih ; Sharif-Askari, Narjes Saheb ; Hamid, Qutayba ; Hundt, Jennifer

OBJECTIVESevere steroid hyporesponsive asthma is a heterogeneous group of chronic inflammatory diseases characterized by irreversible airflow limitation, hyperresponsiveness, inflammation, and remodelling of the airways. Severe asthmatics account for more than 60% of asthma-related healthcare cost worldwide given they are hyporesponsive to corticosteroids and due to the absence of targeted treatment specifically for the T helper-17 (Th-17) high endotype. Hence, there is a clear unmet need to investigate other treatment options to control patients' symptoms. The role of the NLRP3 inflammasome pathway has been highlighted in the literature to contribute to disease pathogenesis and severity. Interestingly, vitamin D3 is an important regulator of the NLRP3 inflammasome pathway.METHODSUsing house dust mite (HDM) and lipopolysaccharide (LPS), we induced a neutrophilic steroid hyporesponsive asthma mouse model to investigate the effect of vitamin D3 on downregulating the NLRP3 inflammasome pathway and enhancing steroid sensitivity.RESULTSWe showed that calcitriol, the active form of vitamin D3, could downregulate the NLRP3 inflammasome pathway. This was associated with a significant reduction in airway hyperresponsiveness, IL-17 release, neutrophil infiltration, and mucus secretion. Further, calcitriol enhanced steroid sensitivity by inhibiting the expression of GR-β. Mechanistically, calcitriol targeted the NLRP3 inflammasome to ubiquitination.CONCLUSIONSOur research highlights the potential use of calcitriol as a low cost and accessible supplement to ameliorate airway inflammation during severe steroid hyporesponsive asthma.

News (Medical) associated with Calcitriol

19 Sep 2024

·www.prnewswire.com

Kyowa Kirin to Present New Research Spotlighting Global Efforts to Advance Science and Patient Care in X-linked Hypophosphatemia at ASBMR Annual Meeting

One oral presentation and nine posters will shed light on real-world experiences of people living with XLH and the impact of burosumab treatment TOKYO, Sept. 19, 2024 /PRNewswire/ -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE:4151) will share new research that enhances understanding about the use of burosumab in real-world clinical practice as well as the burden of those living with X-linked hypophosphatemia (XLH), a rare genetic metabolic bone disease. The findings will be presented at the American Society for Bone and Mineral Research (ASBMR) 2024 annual meeting, which takes place September 27-30 in Toronto. The oral presentation of "Patient reported outcomes (PRO) from a real-world study of burosumab treatment in adults with X-linked hypophosphatemia in the UK" will be made by Judith Bubbear, MD, Royal National Orthopaedic Hospital (UK) on Saturday, September 28, 11:30 am EDT (Presentation #1022). "As we learn more about XLH and how it impacts people's lives, real-world evidence can provide much-needed insight into potential strategies for managing this debilitating disease," said Angela Williams, PhD, Vice President and Global Head of Health Economics and Outcomes Research at Kyowa Kirin. "The breadth of our ASBMR data reflects our continued focus on advancing the science and understanding of XLH and improving the lives of people living with XLH." ASBMR XLH poster presentations: Real-world impact of burosumab treatment in people living with XLH: "Effectiveness of burosumab versus conventional therapy in adults with X-linked hypophosphatemia in a real-world setting from the XLH Disease Monitoring Program" (Poster#SAT-430) Lead author: Pablo Florenzano, MD, Pontificia Universidad Católica de Chile Poster Session I: Saturday, September 28, 2:15 – 3:45 pm EDT "Biochemical measurements according to age group in burosumab-treated patients with X-linked hypophosphatemia (XLH) in a real-world setting: an analysis of the XLH Disease Monitoring Program" (Poster #Fri-424, #Sun-424) Lead author: Leanne M. Ward, MD, Children's Hospital of Eastern Ontario Welcome Reception/Plenary Poster Session: Friday, Sept. 27, 5:30 – 7:30 pm EDT Poster Session II; Sunday, Sept. 29, 2:15 – 3:45 pm EDT "Symptoms experienced by adolescents living with X-linked hypophosphatemia at the end of skeletal growth (EOSG) treated with burosumab—a mixed methods analysis" (Poster #SAT-445) Lead author: Vrinda Saraff, MD, Birmingham Women's and Children's Hospital (UK) Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT "Efficacy and safety of burosumab in adult Chinese patients with X-linked hypophosphatemic rickets/osteomalacia (XLH): an open-label, multi-center, single-cohort, post-marketing phase IV study" (Poster#SUN-119) Lead author: Wei Liu, MD, Peking Union Medical College Hospital Poster Session II: Sunday, Sept. 29, 2:15 – 3:45 pm EDT "Efficacy and safety of burosumab in pediatric Chinese patients with X-linked hypophosphatemic rickets/osteomalacia (XLH): an open-label, multi-center, single-cohort, post-marketing phase IV study" (Poster#SAT-121) Lead author: Xiaoping Luo, MD, Tongji Medical College, Huazhong University of Science and Technology Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT XLH burden on multiple aspects of patients' lives: "Association between pain medication use and patient-reported outcomes in adults with X-linked hypophosphatemia: an exploratory analysis of a phase 3 study" (Poster #Sat-025) Lead author: Angela Williams, PhD, Kyowa Kirin International Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT "Individuals with hereditary hypophosphatemia are not prone to early death despite significant earlier development of co-morbidities: a retrospective Danish register study" (Poster #Sun-024) Lead author: Signe Sparre Beck-Nielsen, MD, PhD, Aarhus University Hospital (Denmark) Poster Session II: Sunday, Sept. 29, 2:15 – 3:45 pm EDT "X-linked Hypophosphatemia Community Impact Survey: psychosocial health, symptoms, and self-care" (Poster #SAT-LB 543) Lead author: Jill Simmons, MD, Vanderbilt University Medical Center Late Breaking Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT Bridging evidence gaps to improve clinical decision-making in XLH: "Advancing Patient Evidence in XLH (APEX): rational and design of real-world XLH global data unification program" (Poster #SAT-427) Lead author: Maria Luisa Brandi, MD, PhD, FIRMO Foundation, Florence, Italy and the University Vita-Salute San Raffaele, Milan, Italy Poster Session I: Saturday, Sep 28, 2:15-3:45 pm EDT About X-linked hypophosphatemia X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults. In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health. This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine. When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in adults (osteomalacia). In children, XLH typically appears as bowed legs or knock knees. Over time, bone weakening can also lead to bone abnormalities in the legs, delayed growth, and short stature. In adults, XLH may cause osteomalacia, fractures and pseudo-fractures, and hypophosphatemia. About CRYSVITA® (burosumab-twza) Injection CRYSVITA is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, which binds to and inhibits the biological activity of FGF23, the underlying cause of hypophosphatemia in XLH. By blocking FGF23, CRYSVITA helps to restore phosphorus reabsorption in the kidneys and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium. U.S. CRYSVITA Indication CRYSVITA is a fibroblast growth factor (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older. Important Safety Information CONTRAINDICATIONS CRYSVITA is contraindicated: In concomitant use with oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol) due to the risk of hyperphosphatemia. When serum phosphorus is within or above the normal range for age. In patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism. WARNINGS AND PRECAUTIONS Hypersensitivity Hypersensitivity reactions (e.g., rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment. Hyperphosphatemia and Risk of Nephrocalcinosis Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient's serum phosphorus levels. Injection Site Reactions Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment. ADVERSE REACTIONS Pediatric Patients Adverse reactions reported in 10% or more of CRYSVITA-treated pediatric XLH patients across three studies are: pyrexia (55%, 44%, and 62%), injection site reaction (52%, 67%, and 23%), cough (52%), vomiting (41%, 48%, and 46%), pain in extremity (38%, 46%, and 23%), headache (34% and 73%), tooth abscess (34%, 15%, and 23%), dental caries (31%), diarrhea (24%), vitamin D decreased (24%, 37%, and 15%), toothache (23% and 15%), constipation (17%), myalgia (17%), rash (14% and 27%), dizziness (15%), and nausea (10%). Postmarketing experience reported in CRYSVITA-treated pediatric XLH patients: blood phosphorus increased. Adult Patients Adverse reactions reported in more than 5% of CRYSVITA-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain (15%), headache (13%), tooth infection (13%), restless legs syndrome (12%), vitamin D decreased (12%), dizziness (10%), constipation (9%), muscle spasms (7%), and blood phosphorus increased (6%). Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression. USE IN SPECIFIC POPULATIONS There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-844-768-3544. There is no information regarding the presence of CRYSVITA in human milk or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition. PATIENT COUNSELING INFORMATION Advise patients not to use any oral phosphate and/or active vitamin D analog products. Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless legs syndrome induction or worsening of symptoms occur. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to Kyowa Kirin, Inc. at 1-844-768-3544. For important risk and use information, please see the full Prescribing Information for CRYSVITA. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato oncology, and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across the globe. Learn more about Kyowa Kirin at: kyowakirin.com. COR-US-CRY-0049 September 2024 SOURCE Kyowa Kirin WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical Result

13 May 2024

·www.globenewswire.com

Entera Bio to Present Phase 1 Data from First-in-Class, PTH(1-34) Peptide Tablets (EB612) for the Treatment of Hypoparathyroidism at ENDO 2024

JERUSALEM, May 13, 2024 (GLOBE NEWSWIRE) -- Entera Bio Ltd. (NASDAQ: ENTX), (“Entera” or the “Company”) a leader in the development of orally delivered peptides and small therapeutic proteins, today announced that it has been selected to present data for its investigational program EB612 at the Endocrine Society ENDO 2024 Annual Meeting which will be held on June 1-4, 2024 in Boston, Massachusetts. The EB612 program is being developed by Entera to provide the first oral PTH(1-34) tablet peptide replacement therapy for patients suffering from hypoparathyroidism. Hypoparathyroidism is characterized by deficient PTH production, hypocalcemia and hyperphosphatemia. Standard treatments include several daily administrations of high dose oral calcium supplements and calcitriol (or analogs), which are often associated with ectopic calcification, including nephrocalcinosis and renal failure. Late stage investigational PTH replacement treatments include TransCon PTH (palopegteriparatide) by Ascendis Pharma A/S and eneboparatide (AZP-3601) by Amolyt Pharma (pending acquisition by AstraZeneca). Both these modalities require patients to administer injections every day. The data from this Phase 1 study include PK and early PD results from the application of a new generation of Entera’s N-Tab™ technology platform with an unmodified PTH(1-34) peptide. One of the objectives of this study was to test the new platform’s ability to reduce the frequency of daily administration of EB612. Entera previously published positive Phase 2a study results in 19 hypoparathyroid patients using a QID regimen (Fraser, JBMR 2021). Abstract Title: Phase 1 Study Results Of EB612, A First-in-Class Oral PTH(1-34) Analog For The Treatment Of Hypoparathyroidism Presentation Number: SAT-758 Presentation Type: Late-Breaking Poster Presentations: Day 1 Session: Poster Session P106 Session Date/Time: Saturday, June 1, 2024, 12:15 pm – 1:45 pm The abstract can be found on the ENDO 2024 website: https://www.abstractsonline.com/pp8/#!/11016/presentation/9825 About Entera Bio Entera is a clinical stage company focused on developing oral peptide or protein replacement therapies for significant unmet medical needs where an oral tablet form holds the potential to transform the standard of care. The Company leverages on a disruptive and proprietary technology platform (N-Tab™) and its pipeline includes five differentiated, first-in-class oral peptide programs, expected to enter the clinic (Phase 1 to Phase 3) by 2025. The Company’s most advanced product candidate, EB613 (oral PTH(1-34)), is being developed as the first oral, osteoanabolic (bone building) once-daily tablet treatment for post-menopausal women with low BMD and high-risk osteoporosis, with no prior fracture. A placebo controlled, dose ranging Phase 2 study of EB613 tablets (n= 161) met primary (PD/bone turnover biomarker) and secondary endpoints (BMD). Entera is preparing to initiate a Phase 3 registrational study for EB613 pursuant to the FDA’s qualification of a quantitative BMD endpoint which is expected to occur by January 2025. The EB612 program is being developed as the first oral PTH(1-34) tablet peptide replacement therapy for hypoparathyroidism. Entera is also developing the first oral oxyntomodulin, a dual targeted GLP1/glucagon peptide, in tablet form for the treatment of obesity; and first oral GLP-2 peptide tablet as an injection-free alternative for patients suffering from rare malabsorption conditions such as short bowel syndrome in collaboration with OPKO Health. For more information on Entera Bio, visit www.enterabio.com or follow us on LinkedIn, Twitter, Facebook, Instagram. Cautionary Statement Regarding Forward Looking Statements Various statements in this press release are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements (other than statements of historical facts) in this press release regarding our prospects, plans, financial position, business strategy and expected financial and operational results may constitute forward-looking statements. Words such as, but not limited to, “anticipate,” “believe,” “can,” “could,” “expect,” “estimate,” “design,” “goal,” “intend,” “may,” “might,” “objective,” “plan,” “predict,” “project,” “target,” “likely,” “should,” “will,” and “would,” or the negative of these terms and similar expressions or words, identify forward-looking statements. Forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Forward-looking statements should not be read as a guarantee of future performance or results and may not be accurate indications of when such performance or results will be achieved. Important factors that could cause actual results to differ materially from those reflected in Entera’s forward-looking statements include, among others: changes in the interpretation of clinical data; results of our clinical trials; the FDA’s interpretation and review of our results from and analysis of our clinical trials; unexpected changes in our ongoing and planned preclinical development and clinical trials, the timing of and our ability to make regulatory filings and obtain and maintain regulatory approvals for our product candidates; the potential disruption and delay of manufacturing supply chains; loss of available workforce resources, either by Entera or its collaboration and laboratory partners; impacts to research and development or clinical activities that Entera may be contractually obligated to provide; overall regulatory timelines; the size and growth of the potential markets for our product candidates; the scope, progress and costs of developing Entera’s product candidates; Entera’s reliance on third parties to conduct its clinical trials; Entera’s expectations regarding licensing, business transactions and strategic collaborations; Entera’s operation as a development stage company with limited operating history; Entera’s ability to continue as a going concern absent access to sources of liquidity; Entera’s ability to obtain and maintain regulatory approval for any of its product candidates; Entera’s ability to comply with Nasdaq’s minimum listing standards and other matters related to compliance with the requirements of being a public company in the United States; Entera’s intellectual property position and its ability to protect its intellectual property; and other factors that are described in the “Cautionary Statements Regarding Forward-Looking Statements,” “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Entera’s most recent Annual Report on Form 10-K filed with the SEC, as well as the company’s subsequently filed Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. There can be no assurance that the actual results or developments anticipated by Entera will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Entera. Therefore, no assurance can be given that the outcomes stated or implied in such forward-looking statements and estimates will be achieved. Entera cautions investors not to rely on the forward-looking statements Entera makes in this press release. The information in this press release is provided only as of the date of this press release, and Entera undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except to the extent required by law.

Phase 2Phase 1Clinical ResultAcquisitionPhase 3

21 Mar 2024

·synapse.patsnap.com

GPCR Targets and Pipeline at the JP Morgan Healthcare Conference 2024 (4)

This article will continue to introduce the GPCR (G Protein-Coupled Receptor) targets and pipelines that garnered attention for Chugai, UCB, Ascendis and BridgeBio at the JP Morgan Healthcare Conference 2024.For more details, please follow the link below. GPCR Targets and Pipeline at the JP Morgan Healthcare Conference 2024 (1)GPCR Targets and Pipeline at the JP Morgan Healthcare Conference 2024 (2)GPCR Targets and Pipeline at the JP Morgan Healthcare Conference 2024 (3)GPCR Targets and Pipeline at the JP Morgan Healthcare Conference 2024 (5)Chugai in JPM 2024Chugai Pharmaceutical is a leading Japanese biopharmaceutical company, established in 1925 with its headquarters in Tokyo. As a member of the Roche Group, Chugai strives to bring Roche's extensive pipeline to the Japanese market, while simultaneously taking Chugai's rich portfolio of innovative drug research and development achievements to the global stage. Chugai is dedicated to the research, development, and commercialization of innovative pharmaceuticals, achieving significant advancements particularly in the fields of oncology, immunology, orthopedics, and neuroscience. The company boasts robust internal R&D capabilities and actively collaborates with other global leading research institutions to undertake comprehensive drug development activities, from early-stage discovery to late-phase clinical trials. Through the formation of strategic alliances, Chugai has realized extraordinary success. From 2001 to 2022, the company's operating profit increased 17-fold, with revenue growing 6-fold. Chugai delivers four joint Sino-foreign products to the global market through Roche and continues to launch and infiltrate markets with proprietary products (Crovalimab, nemolizumab, and orforglipron). Chugai boasts an extensive pipeline of original research, with over 20 antibody drugs, cell therapy and gene therapy products in the drug discovery and preclinical research stages, 9 small molecule drugs, 29 mid-sized molecule drugs, 13 drugs in clinical stages, and 8 marketed drugs. Chugai possesses robust R&D capabilities, and guided by the twin principles of technology-driven and quality-centered approaches, the company has established a range of unique proprietary drug molecules in various categories, including antibodies, small molecules, and cyclic peptide drugs. In the field of antibodies, Chugai possesses 14 patented technologies. Chugai's mid-sized cyclic peptide molecules exhibit a range of advantageous characteristics, including oral bioavailability, the ability to target intracellular targets, and high-affinity binding. Orforglipron (OWL833, LY3502970) is a small molecule GLP-1R agonist developed by Chugai Pharmaceuticals. In 2018, Eli Lilly and Company signed a collaboration agreement with Chugai, paying a $50 million upfront payment to acquire the rights for the clinical development of Orforglipron for the treatment of type 2 diabetes. Between August and September of 2023, Eli Lilly published four clinical research trial data articles sequentially in "The Lancet," "The New England Journal of Medicine," and "Diabetes, Obesity and Metabolism," presenting the results of phase 1a, 1b, and 2 trials of Orforglipron for the treatment of type 2 diabetes and obesity. UCB in JPM 2024UCB Inc. is a global biopharmaceutical enterprise dedicated to developing innovative drugs for the treatment of serious diseases in the fields of immunology and neuroscience. The company's goal is to become the 'patient-preferred' leader in the biopharmaceutical sector by creating patient value for specific communities through unique outcomes and the ultimate experience, and to improve their lives as much as possible. In 2023, the company's blockbuster product CIMZIA achieved its sales peak target of 2 billion euros ahead of schedule, and with a differentiated advantage, its sales growth outperformed the anti-TNF market. It is anticipated that there will be no competition from biosimilars until before 2027. Additionally, five products, including EVENITY®, BIMZELX®, FINTEPLA®, RYSTIGGO®, and ZILBRYSQ®, may possess sales growth momentum for up to ten years. UCB has announced several R&D pipeline candidates that are expected to reach significant clinical data milestones in 2024. Fenfluramine Hydrochloride is a multi-target small molecule drug developed by Zogenix. Its mechanism of action functions as an agonist for 5-HT1D receptors, 5-HT2A receptors, and 5-HT2C receptors, among others. The medication was approved for marketing in 2020 for the treatment of Dravet syndrome and epilepsy. In 2022, UCB announced the acquisition of Zogenix at an approximate total cost of 1.9 billion US dollars. Ascendis in JPM 2024Ascendis Pharma is a global biopharmaceutical company focused on developing innovative and differentiated therapies for rare diseases, using its proprietary TransCon (Transient Conjugation) technology platform to design and manufacture drugs. This platform creates compounds that are temporarily bound (or "transiently conjugated"), allowing for precise control over the drug's release behavior in the body. The TransCon technology enables the activation of the drug after it enters the body, with the release of the active therapeutic ingredient at predetermined times and locations. This technology offers new treatment options for various diseases, especially for patients with rare diseases who require precise drug exposure to achieve optimal therapeutic effects and minimize the risk of side effects. The company's leading endocrinology rare disease products, including TransCon hGH and TransCon PTH, have been approved in major markets. Hypoparathyroidism is an endocrine disorder caused by insufficient or absent secretion of parathyroid hormone (PTH), which can affect multiple organs. Under normal physiological conditions, PTH and its downstream hormone calcitriol are the main regulators of calcium and phosphate, acting on bones, kidneys, and intestines. Hypoparathyroidism can lead to hypocalcemia and hyperphosphatemia. TransCon PTH is an investigational prodrug of PTH hormone originally developed by Ascendis Pharma that is administered via a once-daily injection to provide sustained release of active PTH. It is currently under development for the treatment of adult hypoparathyroidism. This medication consists of the parent drug PTH(1-34) transiently linked to a branched methoxy polyethylene glycol (mPEG) carrier using a proprietary linker. The drug is administered through a once-daily subcutaneous injection, ensuring stable PTH levels within a normal physiological range over 24 hours. By activating downstream PTH1R receptors, it modulates function. BridgeBio in JPM 2024BridgeBio is a next-generation biotechnology company specializing in the treatment of genetic diseases, dedicated to providing innovative therapies to over 450 million patients worldwide suffering from genetic disorders. The company's operations encompass every stage from early research and development to commercialization, with a deep focus on drug development for rare disease areas that have market potential exceeding one billion US dollars. One of BridgeBio's key products currently in development is acoramidis, intended for the treatment of ATTR-CM (transthyretin amyloidosis cardiomyopathy). BridgeBio’s R&D engine is specifically designed for genetic medicines, featuring efficiency, low cost, and rapid decision-making capabilities that allow the advancement of new drug clinical trial applications (IND) at a cost of less than 10 million US dollars, and achieving IND filing in under three years. The company has significant experience in clinical trials, operating in more than 20 trials globally, across 25 countries and over 500 sites, with a particular focus on collaborations with key pediatric hospitals. According to the Phase 3 clinical trial data released by BridgeBio, patients treated with acoramidis demonstrated an unprecedented improvement in absolute survival rates, meaning that patients lived longer and had a significant reduction in hospitalization needs due to the disease, approaching the level seen in healthy individuals of the same age. Additionally, acoramidis showed early divergence from the control group in cardiovascular-related mortality and heart failure composite outcomes within just three months, which is the earliest known time point for observing treatment effects to date. The company's clinical candidate drug Encaleret is indicated for the treatment of Autosomal Dominant Hypocalcemia type 1 (ADH1). This is a rare genetic disorder caused by gain-of-function mutations in the calcium-sensing receptor (CaSR) gene, leading to dysregulated secretion of parathyroid hormone (PTH) and resulting in hypocalcemia. Encaleret sulfate is a product that acts as an antagonist targeting the CaSR receptor. As a potential "first-in-class" disease-modifying therapeutic agent, it is designed to restore physiological mineral homeostasis, assisting ADH1 patients in achieving normal levels of serum calcium and urinary calcium.

100 Deals associated with Calcitriol

External Link

KEGG	Wiki	ATC	Drug Bank
D00129	Calcitriol	D05AX03 A11CC04	DB00136

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Plaque psoriasis	United States	Galderma Laboratories LP	23 Jan 2009
Glucocorticoid-induced osteoporosis	Australia	Atnahs Pharma Australia Pty Ltd.	18 Mar 2003
Hypocalcemia	Australia	Atnahs Pharma Australia Pty Ltd.	18 Mar 2003
Chronic Kidney Disease-Mineral and Bone Disorder	China	Roche Pharma AG	17 Sep 1999
Osteoporosis, Postmenopausal	China	Roche Pharma AG	17 Sep 1999
Bone Diseases, Metabolic	United States	Esjay Pharma LLC	20 Nov 1998
Osteoporosis	Japan	Chugai Pharmaceutical Co., Ltd.	11 Sep 1989
Vitamin D Deficiency	Japan	Chugai Pharmaceutical Co., Ltd.	05 Nov 1985
Hyperparathyroidism, Secondary	United States	Esjay Pharma LLC	17 Aug 1978
Kidney Failure, Chronic	United States	Esjay Pharma LLC	17 Aug 1978

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Osteoporosis	NDA/BLA	China	Hainan Sihuan Pharmaceutical Co. Ltd.	11 Feb 2022
Osteoporosis	NDA/BLA	China	Strides Pharma Science Ltd.	11 Feb 2022
Castration-Resistant Prostatic Cancer	Phase 1	Romania	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 1	Czechia	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 1	Hungary	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 1	Puerto Rico	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 1	Slovakia	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 1	Montenegro	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 1	United States	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 1	Canada	Novacea, Inc.	01 Jan 2006

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02224144 (CTgov)	Phase 2	Bone Diseases, Metabolic \| Vascular Calcification \| Kidney Failure, Chronic \| Fractures, Bone	61	Calcitriol+Vitamin D3 (Vitamin D3 Plus Calcitriol)	scppfkbpsu(zpfxnozxcv) = yupwxnjwob lgmzdtbfox (bhebldodsv, ssnzdvkdis - ohjfhqiaap) View more	-	07 Mar 2025
				Placebo+Vitamin D3 (Vitamin D3 Plus Placebo)	scppfkbpsu(zpfxnozxcv) = bwuxaobyql lgmzdtbfox (bhebldodsv, pzhvhemvfp - tpghfgwimw) View more
ENDO2024	Not Applicable	Renal Cell Carcinoma calcitriol \| intact parathyroid hormone (PTH) \| PTH-related protein (PTHrP) ... View more	-	Calcitriol-mediated hypercalcemia	dtkqeedjze(fbzeiaxcxk) = jrzikdkcvl uwvsqtpokt (zhqankrqdw ) View more	-	01 Jun 2024
ENDO2024	Not Applicable	Hypoparathyroidism PTH mutation	-	Teriparatide 20 mcg SC daily	(cojmxksinz) = nvnoywporn kfranikivg (kzdykpsich ) View more	Positive	01 Jun 2024
				Calcitriol and calcium carbonate	(cojmxksinz) = zruvytkusc kfranikivg (kzdykpsich ) View more
WCO_IOF_ESCEO2024	Not Applicable	-	61	Calcitriol 0.5 mcg	mbpjjpqljl(oclwqxmjgc) = Hypercalcemia occurred in 39% and 3% in the calcitriol and placebo groups respectively vjkysjmfxp (kwsjdhvtcg )	Negative	11 Apr 2024
				Placebo
ISRCTN17354692 (PRECAL study, ENDO2023)	Phase 2	Diabetes Mellitus, Type 1 Insulin auto-antibodies \| anti-tyrosinase abs \| anti-glutamic decarboxylase abs ... View more	50	Calcitriol	jixkpjlegb(bvaxuczmzo) = did not progress to T1D cuebwrjicx (geudobzaab ) View more	Positive	05 Oct 2023
SAT258 (ENDO2023)	Not Applicable	Adult T-Cell Leukemia-Lymphoma HTLV-1 \| PTHrP	1	Denosumab 60 mg	(avqzlrvdoz) = tlbwidnfor rnqncijozm (tvydyjsmfe ) View more	Positive	05 Oct 2023
				Denosumab 120 mg	goddonnkru(wysdeuhckv) = dizpbdtuqj rhglkoxzet (keytbbzisy )
EHA2023	Not Applicable	Juvenile Myelomonocytic Leukemia	29	Tretinoin	aggsczjpbl(umzfsmxvfn) = rdgeyizimu yzdlhjwcti (ibpponiyfi )	-	08 Jun 2023
				Calcitriol	aggsczjpbl(umzfsmxvfn) = kpoyqiuzxx yzdlhjwcti (ibpponiyfi )
ESPE2022	Not Applicable	Familial Hypophosphatemic Rickets PHEX gene mutation	15	Burosumab	(hxnafwkdmx) = reported in one patient jhzqtndoja (tawzeztgcz ) View more	-	15 Sep 2022
				Conventional treatment (inorganic oral phosphate salts and calcitriol or alphacalcidol)
NCT00763555 (CTgov)	Phase 2	Plaque psoriasis	88	CD 2027 (Calcitriol 3 mcg/g Spray)	hewlyftxdf(rroeqbzobd) = flrrzpshge ddocmpqvkp (qkxahukjkg, ndiwhobjrt - ljwrgtnono) View more	-	23 Aug 2022
				Calcitriol Vehicle (Calcitriol Vehicle)	hewlyftxdf(rroeqbzobd) = xwewqmpoii ddocmpqvkp (qkxahukjkg, gykcnirpze - cibqvkwclo) View more
MO803 (ERA2022)	Not Applicable	Hyperparathyroidism, Secondary	-	Paricalcitol	rnhesdstxq(xwvfngkpqr) = gwvrawsvso ivpogdxdxw (polsaxltif )	Positive	03 May 2022
				Calcitriol	rnhesdstxq(xwvfngkpqr) = jitwczpdgq ivpogdxdxw (polsaxltif )

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